Tolerability and Efficacy of a Combination of Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

This study is currently recruiting participants.
Verified May 2013 by Brigham and Women's Hospital
Sponsor:
Information provided by (Responsible Party):
Rodney H. Falk, MD, Brigham and Women's Hospital
ClinicalTrials.gov Identifier:
NCT01855360
First received: March 12, 2013
Last updated: November 19, 2013
Last verified: May 2013

March 12, 2013
November 19, 2013
June 2013
September 2015   (final data collection date for primary outcome measure)
The rate of progression of transthyretin cardiac amyloidosis, as measured by changes in strain echocardiography, in a group of patients taking a combination of doxycycline and TUDCA. [ Time Frame: Time Frame: * (FDAAA) outcome measure is assessed every 6 months by serial echocardiography, with final measurement 18 months after enrollment. ] [ Designated as safety issue: No ]
Outcome measure, namely changes in longitudinal echocardiographic strain, will be compared to previously determined changes derived from a cohort of patients with TTR cardiac amyloidosis who were not receiving specific therapy for amyloid deposition.
Same as current
Complete list of historical versions of study NCT01855360 on ClinicalTrials.gov Archive Site
  • Number of patients with adverse events to the medications over the period of therapy (18 months) [ Time Frame: 18 months ] [ Designated as safety issue: Yes ]
    Measurements of kidney function (BUN and creatinine) and blood counts will be done every 3 months.
  • To evaluate general and health related quality of life (QoL) in SSA and ATTR subjects. [ Time Frame: Measurements made every 6 months for 18 months. ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Tolerability and Efficacy of a Combination of Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy
An 18 Month, Open Label Study of the Tolerability and Efficacy of a Combination of Doxycycline and Tauroursodeoxycholic Acid (TUDCA) in Patients With Transthyretin Amyloid Cardiomyopathy.

The objective of the study is to determine whether the combination of the bile acid TUDCA, and doxycycline will slow the progression of familial and senile amyloidosis.

Not Provided
Interventional
Phase 1
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Amyloidosis; Heart (Manifestation)
  • Senile Cardiac Amyloidosis
Drug: Tauroursodeoxycholic Acid and Doxycycline
Experimental: TUDCA and Doxycycline
TUDCA taken orally, 250 mg three times daily. Doxycycline taken orally, 100 mg twice daily
Intervention: Drug: Tauroursodeoxycholic Acid and Doxycycline
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
40
Not Provided
September 2015   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Documented transthyretin cardiac amyloidosis by biopsy and staining using immunohistochemistry or mass spectrometry
  • Echocardiographic appearance of left ventricular wall thickness of 13mm or more in the absence of hypertensive heart disease
  • Confirmed ATTR or SSA by genetic testing
  • Age 18-90
  • Male or non-pregnant, non-lactating females
  • Willingness to return to the treatment center for follow-up

Exclusion Criteria:

  • Prior liver transplantation or liver transplantation anticipated in less than 6 months
  • Alanine Transaminase and/or Aspartate Transaminase ≥2 x upper normal limit (UNL)
  • Alkaline Phosphatase ≥2 x UNL
  • Creatinine clearance <20 mL/min
  • Any other lab values that in the opinion of the investigator might place the subject at unacceptable risk for participation in the study
  • History of poor compliance
  • History of hypersensitivity to any of the ingredients of the study therapies
  • Any investigational drug within 4 weeks prior to study entry or during the study
  • Current use of diflunisal for therapy of amyloidosis
Both
18 Years to 90 Years
No
Contact: Rodney H Falk, MD 617 421 6094 rfalk@partners.org
United States
 
NCT01855360
BWHAMY1
No
Rodney H. Falk, MD, Brigham and Women's Hospital
Brigham and Women's Hospital
Not Provided
Principal Investigator: Rodney H Falk, MD Brigham and Women's Hospital, Boston MA
Brigham and Women's Hospital
May 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP